APL2 for FSGS

Not yet recruiting at 1 trial location

Overseen By Apellis Clinical Trial Information Line

Age: 18+

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Apellis Pharmaceuticals, Inc.

Must not be taking: Rituximab, Belimumab, Anticomplement

Disqualifiers: Diabetes, Kidney transplant, HIV, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test the safety and effectiveness of a treatment called APL2 for individuals with FSGS, a kidney condition that causes excess protein in the urine and potential kidney damage. Participants in Phase 2 will receive APL2 infusions twice a week. Phase 3 will compare APL2 against a placebo (a substance with no active drug) to assess its true benefits. The trial is open to adults and, in some cases, teenagers diagnosed with FSGS through a kidney biopsy or genetic testing, who have stable treatment plans and high levels of protein in their urine. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, offering a chance to contribute to important medical advancements.

Do I have to stop taking my current medications for the trial?

The trial requires that you have been on a stable treatment plan for FSGS for at least 12 weeks before joining, with no changes expected. This suggests you should continue your current medications without changes.

Is there any evidence suggesting that APL2 is likely to be safe for humans?

Research has shown that APL2, a treatment for FSGS, has been tested in people before. APL2 works by blocking a part of the immune system, helping to control it. Past studies have demonstrated that APL2 is safe, and patients have generally tolerated the treatment well, with few serious side effects.

The treatment involves infusions under the skin, administered twice a week. Patients usually find this method easy to manage. While all treatments can have side effects, the safety data for APL2 appears promising, especially as the trial progresses into later stages, indicating positive earlier results.

Discussing potential risks with the study team is always important, but current evidence suggests APL2 is quite safe to use. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for FSGS?

Researchers are excited about APL2 for FSGS because it offers a novel approach by targeting the complement system, which plays a role in inflammation and kidney damage. Unlike traditional treatments like steroids and immunosuppressants that broadly suppress the immune system, APL2 specifically inhibits the C3 protein, potentially reducing side effects and providing more precise treatment. Additionally, APL2 is administered via subcutaneous infusions, which could offer more consistent dosing and convenience compared to oral medications. This new mechanism and delivery method make APL2 an appealing option with the potential for improved outcomes for patients with FSGS.

What evidence suggests that APL2 might be an effective treatment for FSGS?

Research shows that APL2 is designed to block a protein called complement C3, which might help reduce kidney damage in FSGS, a type of kidney disease. Early results suggest that this could effectively slow down or stop the disease's progression. Studies have found that targeting complement C3 may help prevent the kidneys from leaking proteins, a major issue in FSGS. Although direct human data on APL2 for FSGS is still being gathered, its mechanism appears promising. Participants in this trial will receive either APL2 or a placebo to further evaluate its effectiveness. ^{1 3 6 7 8}

Are You a Good Fit for This Trial?

Adults and adolescents with Focal Segmental Glomerulosclerosis (FSGS) can join this study. The trial has two parts: first, a phase where everyone gets the drug APL2 to see if it's safe, then a second part where participants are randomly chosen to either continue with APL2 or switch to a placebo without knowing which one they're getting.

Inclusion Criteria

Estimated glomerular filtration rate (eGFR) ≥25 mL/min/1.73 m2

I am 18 years or older, or between 12-17 where approved, and can consent to participate.

My weight is between 30 kg and 100 kg.

Exclusion Criteria

Hypersensitivity to APL2 or to any of the excipients

I have type 1 diabetes or my type 2 diabetes is not well-controlled.

I have been treated with APL2 before.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Phase 2 Treatment

Participants receive twice-weekly subcutaneous infusions of APL2 in a single-arm, open-label study

12 weeks

Twice-weekly visits for infusions

Phase 3 Treatment

Participants are randomized to receive either APL2 or placebo in a double-blind, placebo-controlled study

52 weeks

Twice-weekly visits for infusions

Follow-up

Participants are monitored for safety and effectiveness after treatment

52 weeks

What Are the Treatments Tested in This Trial?

Interventions

• APL2

Trial Overview The trial is testing APL2, an experimental medication given by injection under the skin twice weekly for its effectiveness in treating FSGS. It compares results from patients on APL2 against those on a placebo to determine if there's any improvement in their condition.

How Is the Trial Designed?

Group I: Phase 3 - APL2 Experimental Treatment 1 Intervention

Adults: Sub-cutaneous infusions of APL2 (1080 mg / 20mL) twice weekly Adolescents: ≥50 Kg: Sub-cutaneous infusions of APL2 (1080 mg / 20mL) twice weekly 35 to \<50kg: First sub-cutaneous infusion of 648mg (12 mL) followed by 810mg (15 mL) every infusion thereafter (i.e. twice weekly) 30 to \<35kg: First \& Second sub-cutaneous infusion of 540mg (10mL) followed by 648mg (12 mL) every infusion thereafter (twice weekly)

Group II: Phase 2 - APL2 Experimental Treatment 1 Intervention

Sub-cutaneous infusions of APL2 (1080 mg / 20mL) twice weekly

Group III: Phase 3 - Placebo Placebo Group 1 Intervention

Subcutaneous infusions of a sterile solution, twice-weekly, and equivalent in volume to the active arm based on participant's age and weight

Who Is Running the Clinical Trial?

Apellis Pharmaceuticals, Inc.

Lead Sponsor

Trials

26

Recruited

4,300+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT07213960

NCT07213960 | A Sequential Phase 2/3 Study of APL2 in ... See Delay Results Type in the Results Data ... Efficacy and Safety of APL2 in Adults and Adolescents With Focal Segmental Glomerulosclerosis.

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/41055958/

Treatment Response Rates and Kidney Outcomes among ... Results: Among 228 patients treated with IS, 55% achieved remission (12% CR, 43% PR), with relapse rates of 63% and 75% by two years. Median ...

3. tipranks.com tipranks.com/news/company-announcements/apellis-pharmaceuticals-advances-apl2-study-for-rare-kidney-disease

Apellis Pharmaceuticals Advances APL2 Study for Rare ... APL2 is designed to inhibit complement C3, potentially reducing kidney damage in FSGS patients. The study also includes a placebo comparator for ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11812565/

Advances in Focal Segmental Glomerulosclerosis ... 9 However, approximately 32% of pFSGS patients experience a recurrence of disease in the early period following renal transplantation, with a ...

5. khi.asn-online.org khi.asn-online.org/uploads/1-s2.0-S0272638624010382-main.pdf

Proteinuria as an End Point in Clinical Trials of Focal ... 47 Data on long-term kidney outcomes in patients who participated in the trial were not systematically captured. The DUPLEX Study, investigating sparsentan ...

6. withpower.com withpower.com/trial/phase-2-and-3-fsgs-11-2025-d03ee

APL2 for FSGS · Info for Participants This is a sequential phase 2/3 study to evaluate the efficacy and safety of twice-weekly subcutaneous (SC) infusions of APL2 in patients ...

7. adisinsight.springer.com adisinsight.springer.com/trials/700379488

A Sequential Phase 2/3, Single-Arm, Open-Label Study in ... This is a sequential phase 2/3 study to evaluate the efficacy and safety of twice-weekly subcutaneous (SC) infusions of APL2 in patients diagnosed with ...

8. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC3306824/

Clinical trial of focal segmental glomerulosclerosis in children ... One complete remission and six partial remissions (urine protein/creatinine (Up/c) <2 grams per grams (g/g)) were observed, yielding a combined response of 47%, ...